December 10, 2017
by inesp.alves
8 Comments
BioMarin Pharmaceutical sent to patient organizations an update of the clinical development program for BMN-111. The current status of the achondroplasia clinical programme is described in the document below. BioMarin’s investigational therapy for achondroplasia, BMN 111, is currently under investigation … Continue reading
October 20, 2017
by inesp.alves
0 comments
The following document, shared by BioMarin in September 2017, is a clarification on the ongoing clinical trials for achondroplasia and was released for associations and families: BioMarin Achondroplasia Programme Update 20Sept17 It is important to mention that families interested in … Continue reading
June 3, 2017
by inesp.alves
1 Comment
The “Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia” is a prospective observational study. This study’s code name is 111-901. The aim of this study is to collect systematic growth measurements of the children in order to collect data … Continue reading
May 12, 2017
by inesp.alves
4 Comments
The following information is fully available at the European Union Clinical Trials Register. Some sections will be here highlighted: 1. Full title of the trial A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN … Continue reading
November 23, 2016
by inesp.alves
1 Comment
A rare disease encompasses any life-threatening or chronically debilitating disorder or condition which, as the name suggests, is uncommon in the general population. Rare diseases typically exhibit a high level of symptom complexity and as a result they very frequently require … Continue reading
November 7, 2016
by inesp.alves
1 Comment
In a collaboration initiative between ALPE Foundation, FIMABIS and Dr. Felipe Luna, Head of Orthopedic Surgery and Traumatology at Málaga Central Hospital, Spain, the Second Conference of Malaga Achondroplasia Unit “Around achondroplasia – research for families” will be held in … Continue reading
October 20, 2016
by inesp.alves
3 Comments
BioMarin presented on the 19th October 2016 a press release on the data for Vosoritide in phase 2, during the ASHG 2016 meeting. There is no overall novelty in this latest press release (19th Oct 2016) when comparing to the previous press … Continue reading
May 17, 2016
by inesp.alves
0 comments
LP Doc Talk is a project of Growing Stronger, a foundation based in S. Francisco, California and founded by Amer and Munira Haider, parents of Ahmin,a boy now with 7 years-old, born with achondroplasia. When I started to search about achondroplasia, I … Continue reading
May 13, 2016
by inesp.alves
3 Comments
Study 111-202 A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children with Achondroplasia (ACH) This study is enrolling participants by invitation only Estimated Enrollment: 46 Study start date: January 2014 Estimated Study Completion … Continue reading
April 14, 2016
by inesp.alves
1 Comment
Growing Stronger provides LP Doc talk, a series of online, live conversations with world-leading doctors and other experts focused on dwarfism. You can join live online for each session or visit the archived recordings anytime thereafter. This is a extremely … Continue reading